Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

Autor: Germain, Dominique P., Nicholls, Kathy, Giugliani, Roberto, Bichet, Daniel G., Hughes, Derralynn A., Barisoni, Laura M., Colvin, Robert B., Jennette, J. Charles, Skuban, Nina, Castelli, Jeffrey P., Benjamin, Elfrida, Barth, Jay A., Viereck, Christopher
Zdroj: In Genetics in Medicine September 2019 21(9):1987-1997
Databáze: ScienceDirect
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